Systemic Evaluation of Chimeric LNA/2′OMe Steric Blockers for Myotonic Dystrophy Type 1 Therapy

Melina Christou,Jesper Wengel,Kleitos Sokratous,Kyriacos Kyriacou,Georgios Nikolaou,Leonidas A. Phylactou,N. Mastroyiannopoulos

Systemic Evaluation of Chimeric LNA/2′OMe Steric Blockers for Myotonic Dystrophy Type 1 Therapy

2019

Melina Christou
Jesper Wengel
Kleitos Sokratous
Kyriacos Kyriacou
Georgios Nikolaou
Leonidas A. Phylactou
N. Mastroyiannopoulos

Myotonic dystrophy type 1 (DM1) is a dominantly inherited, multisystemic disorder characterized clinically by delayed muscle relaxation and weakness. The disease is caused by a CTG repeat expansion...

Keywords:

Disease
Endocrinology
Weakness
Steric effects
Internal medicine
Myotonic dystrophy
Biology
muscle relaxation
Molecular biology
Trinucleotide repeat expansion
In vivo
antisense oligonucleotide
ctg repeat

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